Tuesday, June 24, 2014

Big News From Vertex

The Phase 3 results for the Kalydeco/VX-809 combo are in, and the final step is submitting everything for FDA approval! For the approximate 50% of the CF population that is homozygous F508del (or those that have 2 copies of that mutation), this is HUGE news. Heck, it's pretty big news for the rest of us as well.

It's going to be a while before the heterozygous F508del studies reach this point, and possibly longer yet before VX-661 gets there, but this is exciting news! It's not a cure, and we still need to look for a cure. But this drug has the potential to dramatically improve life expectancy and quality of life in CF patients who can get access to the drug.

Personally, I'm remaining cautiously optimistic. There are a lot of good things with Kalydeco, and I'm excited to read the phase 3 study results in-depth to see the positives that came out of it. At the same time, I've also read some of the negatives that are happening, and I really want to know what the long-term effects are going to be. I'm fully aware that we won't know the long-term effects for a while, but it's still something I can't help but think about.

In any case, it's an exciting day for the CF community, and I'm hopeful that the FDA will expedite the approval process so we can get this into the hands of patients as soon as possible!